Combined umbilical cord blood and bone marrow from HLA-identical sibling donors for hematopoietic stem cell transplantation in children with hemoglobinopathies

Pediatr Blood Cancer. 2014 Sep;61(9):1690-4. doi: 10.1002/pbc.25085. Epub 2014 May 7.

Abstract

Background: It is well established that umbilical cord blood and bone marrow are biologically different stem cell sources.

Patients and methods: We analyzed the feasibility and outcome of hematopoietic stem cell transplantation (HSCT) in 13 children (median age 5.9 years) with hemoglobinopathies after the co- infusion of cord blood (CB) and bone marrow (BM) from the same human leucocyte antigen (HLA) identical sibling donor. We also compared outcomes of children with co-transplantation to outcomes in children with hemoglobinopathies who had received a BM (n = 21) or CB (n = 22) transplant alone.

Results: Compared to CB transplant (CBT) recipients, the co-transplant group had more rapid neutrophil (17 vs. 25 days, P = 0.013) and platelet (29 vs. 48 days, P = 0.009) recovery and less transplant related mortality. Patients who received a co-transplant had a lower incidence of ≥ grade II acute (0% vs. 26.3%) and chronic (0% vs. 21%) graft versus host disease (GVHD) compared to BM transplant (BMT) recipients (P = 0.055 and 0.045, respectively). With a median follow-up of >60 months in each treatment group, the 5-year probability of event free survival (EFS) was 100% in the co-transplant group, 90% after BMT and 86% after CBT (P = 0.42).

Conclusion: Co-transplantation of CB and BM from HLA-identical sibling donors appears to be a feasible and effective strategy to further optimize outcomes of HSCT for hemoglobinopathies.

Keywords: bone marrow transplant; cord blood transplant; hemoglobinopathy; sickle cell disease; thalassemia.

MeSH terms

  • Adolescent
  • Bone Marrow Transplantation*
  • Child
  • Child, Preschool
  • Cord Blood Stem Cell Transplantation*
  • Feasibility Studies
  • Female
  • Follow-Up Studies
  • Graft Survival
  • HLA Antigens / immunology
  • Hematopoietic Stem Cell Transplantation*
  • Hemoglobinopathies / mortality
  • Hemoglobinopathies / therapy*
  • Histocompatibility Testing
  • Humans
  • Infant
  • Male
  • Prognosis
  • Survival Rate

Substances

  • HLA Antigens